BioMarin’s orphan drug Brineura will cost $702,000-per-year

The treatment for a rare pediatric brain disorder, known as Batten disease, from BioMarin Pharmaceutical Inc. won approval from FDA, however it will have an extraordinary price tag: $702,000 a year and even with discounts it would be one of the most expensive drugs in the world. The drug is called Brineura.

Most of the patients that have this disorder are on federal assistance programs and the price after mandatory government discounts will be $486,000.
Before this treatment got approved there was no available therapy for the Batten disease which leads most patients to die between the ages of eight and 12.

In the past year drug price has been a very much discussed issue.
The drug industry has argued that it needs to be adequately rewarded for the risks and costs that come with developing novel therapies, and have warned that without sufficient reimbursement, investors will stop supporting the industry.

The chairman and CEO JJ Bienaimé said the system will have the final say on whether the sky-high price is fair.

“Obviously, the system will have to determine that,” he told to Jim Cramer on a CNBC interview. But I would say that CLN2 ( Batten disease), the disease that Brineura is being used for, is an absolutely devastating neurodegenerative disorder for which, unfortunately, the patients will lose their life, most of the time by [age] 12.”